FDA leaders highlight new rare‑disease flexibilities and push for broader compassionate use At an FDA virtual town hall during Rare Disease Week, agency leaders and a patient‑advocate described a new 'plausible mechanism' pathway for ultra‑rare conditions, reiterated transparency measures and urged companies to permit compassionate use and expanded access.

FDA leaders unveil groundbreaking changes to speed up access to treatments for ultra-rare diseases, promising hope for patients with limited options.

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